Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B

This study is looking at a new treatment called Hemgenix for men in the UK who have severe or moderately severe haemophilia B. Haemophilia B is a condition where your blood doesn't clot properly. Hemgenix is a type of gene therapy, which aims to help your body make the missing clotting factor it needs. The main goal of the study is to see if Hemgenix is as good as, or better than, the usual treatments you might have for preventing bleeds. Researchers will compare how many bleeding episodes patients have after receiving Hemgenix to what they experienced before, over a period of 52 weeks (about a year), roughly 6 to 18 months after getting the treatment. They will also check the levels of the clotting factor in the blood and how much other medication is needed to control bleeds.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

CSL Behring LLC

Enrolment target

Start

02 Aug 2024

Haemophilia B

What is this study about?

Imagine your body is a factory, and it needs a specific part (a clotting factor, called Factor IX) to make your blood clot properly. If you have haemophilia B, your factory either makes very little or none of this important part. This means you can have unexpected bleeds that are hard to stop.

Currently, many people with haemophilia B have regular injections to replace the missing clotting factor. This study is testing a new way of treating haemophilia B called gene therapy, specifically a treatment named Hemgenix. Instead of replacing the clotting factor regularly, Hemgenix aims to give your body a tiny instruction manual (a gene) to help your own cells produce the Factor IX they need. Think of it like giving your factory a permanent upgrade so it can make the missing part itself.

The main goal of this study is to see if Hemgenix is at least as good as, or even better than, the standard regular treatments you might be currently having to prevent bleeds. The researchers will be carefully counting how many bleeding episodes people have, and checking their blood to see if their Factor IX levels improve after getting Hemgenix. This information will help doctors understand how well this new treatment works and if it could be a good option for people with haemophilia B in the future.

Key takeaways

This study is for adult men with severe or moderately severe haemophilia B.
It's testing a new gene therapy called Hemgenix, given as a single infusion.
The main goal is to see if Hemgenix can reduce bleeding episodes compared to current treatments.
Researchers will monitor Factor IX levels and bleeding rates for about 18 months after treatment.
Participation involves regular clinic visits, blood tests, and recording any bleeds.

Who may be eligible?

To join this study, participants must be adult men, 18 years old or older, who have been diagnosed with either severe or moderately severe haemophilia B. This means you have a significant lack of the Factor IX clotting protein in your blood, making you prone to bleeding.

They are looking for people who can safely take part and who meet specific health requirements determined by the study doctors. For instance, they'll check if you have certain antibodies in your blood that might affect how the gene therapy works.

It's important that you discuss your medical history and current health with the study team to see if this study is right for you. They will explain all the details and answer any questions you have.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult man (18 years or older)?
Have you been diagnosed with severe or moderately severe haemophilia B?
Are you currently receiving treatment or have you received treatment for your haemophilia B?
Are you willing and able to attend regular clinic appointments for about 18 months?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive a single treatment of the study medication, Hemgenix, given as an infusion into your vein. Before and after this treatment, you would have regular visits to the clinic.

These visits will involve various tests, such as blood tests to check your Factor IX levels and to monitor your overall health. You will also keep a record of any bleeding episodes you experience. The study aims to follow participants for over a year (about 18 months) after the treatment to see how well it's working and if it continues to be effective.

Potential risks and benefits

Participating in a study like this might offer the potential benefit of a new, long-lasting treatment that could reduce your bleeding episodes and potentially lower your need for frequent Factor IX injections. However, as with any new treatment, there are potential risks and side effects that the study doctors will explain to you in detail. These could include reactions to the infusion or other effects related to the gene therapy. Your safety and well-being are the top priority, and you are free to withdraw from the study at any time without giving a reason, and it will not affect your usual medical care.

Locations (6)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Belgium
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Unverified
Netherlands
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Unverified
Germany
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Unverified
Ireland
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Unverified
Denmark
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Unverified
Sweden

Common questions

What is haemophilia B?

Haemophilia B is a genetic condition where your blood either doesn't have enough of a clotting protein called Factor IX, or the Factor IX it has doesn't work properly. This means you can bleed for longer after an injury or have spontaneous bleeds.

What is gene therapy?

Gene therapy is a new type of treatment that aims to correct the root cause of a condition by introducing new genetic material into your body's cells. In this case, it's designed to help your body produce its own working Factor IX.

What is Hemgenix?

Hemgenix is the specific gene therapy being tested in this study. It's designed to give your body the instructions it needs to produce Factor IX, which helps your blood clot.

How long will the study last for me?

After you receive the Hemgenix treatment, you would be followed for about 18 months in total, with regular check-ups and blood tests.

What does 'severe or moderately severe' haemophilia B mean?

It refers to how low your Factor IX levels are. People with severe or moderately severe haemophilia B have very low levels of Factor IX, making them more prone to serious bleeding.